DK1414996T3 - Methods for Genetically Modifying Hematopoietic Progenitor Cells and Uses of Modified Cells - Google Patents

Methods for Genetically Modifying Hematopoietic Progenitor Cells and Uses of Modified Cells

Info

Publication number
DK1414996T3
DK1414996T3 DK02746963.4T DK02746963T DK1414996T3 DK 1414996 T3 DK1414996 T3 DK 1414996T3 DK 02746963 T DK02746963 T DK 02746963T DK 1414996 T3 DK1414996 T3 DK 1414996T3
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DK
Denmark
Prior art keywords
cells
methods
hematopoietic progenitor
genetically modifying
modified
Prior art date
Application number
DK02746963.4T
Other languages
Danish (da)
Inventor
Geoffery P Symonds
Rafael Amado
Lun-Quan Sun
Janet Macpherson
Greg Fanning
Wayne Gerlach
Original Assignee
Johnson & Johnson Res Pty Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Johnson & Johnson Res Pty Ltd filed Critical Johnson & Johnson Res Pty Ltd
Application granted granted Critical
Publication of DK1414996T3 publication Critical patent/DK1414996T3/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • C12N15/1132Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses against retroviridae, e.g. HIV
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121Hammerhead
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • AIDS & HIV (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Described are compositions and methods relating to gene therapy, particularly as applied to hematopoietic progenitor (HP) cells, to transduced cells and methods of obtaining them, and to methods of using them to provide prolonged engraftment of modified hematopoietic cells in human subjects. The invention particularly relates to ex vivo gene therapy of HP cells for treatment or prevention of HIV infection.
DK02746963.4T 2001-07-10 2002-07-10 Methods for Genetically Modifying Hematopoietic Progenitor Cells and Uses of Modified Cells DK1414996T3 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US30412701P 2001-07-10 2001-07-10
US30428301P 2001-07-10 2001-07-10
US34348401P 2001-12-21 2001-12-21
US38606302P 2002-06-04 2002-06-04
PCT/US2002/021907 WO2003006691A1 (en) 2001-07-10 2002-07-10 Methods for genetic modification of hematopoietic progenitor cells and uses of the modified cells

Publications (1)

Publication Number Publication Date
DK1414996T3 true DK1414996T3 (en) 2011-03-21

Family

ID=27501852

Family Applications (1)

Application Number Title Priority Date Filing Date
DK02746963.4T DK1414996T3 (en) 2001-07-10 2002-07-10 Methods for Genetically Modifying Hematopoietic Progenitor Cells and Uses of Modified Cells

Country Status (16)

Country Link
US (2) US7345025B2 (en)
EP (2) EP2322618A1 (en)
JP (2) JP5179697B2 (en)
KR (1) KR101015913B1 (en)
CN (2) CN1553963A (en)
AT (1) ATE490322T1 (en)
AU (1) AU2002316640C1 (en)
BR (1) BR0211079A (en)
CA (1) CA2453531A1 (en)
DE (1) DE60238486D1 (en)
DK (1) DK1414996T3 (en)
HK (1) HK1065074A1 (en)
IL (2) IL159637A0 (en)
MX (1) MXPA04000268A (en)
NZ (1) NZ530624A (en)
WO (1) WO2003006691A1 (en)

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JP5946775B2 (en) * 2010-12-01 2016-07-06 日産化学工業株式会社 Method for producing hematopoietic stem cells using pyrazole compounds
DE102010053973A1 (en) 2010-12-09 2012-06-14 Fresenius Medical Care Deutschland Gmbh Medical device with a heater
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US9186449B2 (en) 2011-11-01 2015-11-17 Fresenius Medical Care Holdings, Inc. Dialysis machine support assemblies and related systems and methods
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CN113897398A (en) * 2012-09-07 2022-01-07 再生元制药公司 Genetically modified non-human animals and methods of use thereof
DK3939423T3 (en) * 2012-11-05 2024-05-06 Regeneron Pharma Genetically modified non-human animals and methods of use thereof
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Also Published As

Publication number Publication date
HK1065074A1 (en) 2005-02-08
IL159637A0 (en) 2004-06-01
EP1414996B1 (en) 2010-12-01
KR20040032852A (en) 2004-04-17
JP2005528319A (en) 2005-09-22
CN101926824A (en) 2010-12-29
US7776595B2 (en) 2010-08-17
US20080044394A1 (en) 2008-02-21
BR0211079A (en) 2005-08-30
KR101015913B1 (en) 2011-02-23
NZ530624A (en) 2009-02-28
US7345025B2 (en) 2008-03-18
EP1414996A1 (en) 2004-05-06
EP2322618A1 (en) 2011-05-18
JP2010077125A (en) 2010-04-08
DE60238486D1 (en) 2011-01-13
ATE490322T1 (en) 2010-12-15
CN1553963A (en) 2004-12-08
WO2003006691A1 (en) 2003-01-23
AU2002316640C1 (en) 2009-01-29
MXPA04000268A (en) 2005-03-07
IL159637A (en) 2013-07-31
EP1414996A4 (en) 2007-03-28
CA2453531A1 (en) 2003-01-23
JP5179697B2 (en) 2013-04-10
US20040072771A1 (en) 2004-04-15
AU2002316640B2 (en) 2008-06-12

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